To the best of my knowledge, this is completely new and unplanned. Wonder what it's about?
ODD doesn't show anything. I'm long ZLCS and feel positively about P2, but the orphan drug designation doesn't mean anything about efficacy. The FDA doesn't give the designation based on trial data; only based on the indication that a drug is hoping to treat.
I do agree that $4 by end of year is quite doable with two positive P2 trials.
Rico, do you have OCD? Even after positive news comes out--first SRPT owning the entire DMD pot and then 96-week data--two days later you still want more news. I'm sure on Monday you'll say the same thing even though more news will be released this week at WMS.
No offense, but if you can't provide proof, then your post is nothing more than a mindless, manipulative pump. (And that's coming from someone who thinks results will be positive.)
Gary, both parties are to blame for the entire mess we are in. Fully agree. As for the shutdown, this is on the Republicans; sdijc is right. If they wanted to change Obamacare funding, they had SEVERAL MONTHS before October 1 to do so, and they did nothing. It is law (like sd, I think it's a #$%$ law), and the Supreme Court upheld it. The Tea Party is acting like a bunch of whiny beeotches. Frankly, they're holding the American people hostage with their tactics and it's disgusting. The Tea Party are like the new Joe McCarthy's. Eventually citizens will realize just how nuts these guys are (despite having a few okay ideas).
Christine, have the doctors overseeing any of the boys on eteplirsen written letters to the FDA? If not, it would be great if they would!
I'm referring to primary care physicians, physical therapists, etc, by the way. Maybe even get some teachers, too.
Then I went long and booked more profits. I make a lot of money with SRPT, and I like to post about it here so everyone knows. I also ate Biscuits and Gravy for lunch at a place called Jimmy's Diner. It was delicious. Oh, and last night I drank a really #$%$ beer called Genny Lite that I'd never even heard of. I think it was on par with or below that of Milwaukee's Best.
Not a fan of Obama, but could you please explain how he's a traitor? It's pretty absurd to throw around such titles at people simply because you disagree with them. Anyway, let us know how he's a traitor.
Why do you compulsively post about ZLCS if you have no further desire to get back in? When most people let go of a stock, don't they just put it in the past and move on to new ones? That's what I do.
I'd say including drisa boys in an etep trial arm was the best news that I heard, but for my wallet, I'd go with CMC.
“I became increasingly aware that I could possibly be the mother of the last child to die from Duchenne and the first child to survive it. That was unacceptable to me.”
Way to go, Jenn! Keep on keepin' on!!!! Here's hoping for the best for both Max and Austin. (And Christine's son, Jett, and all other boys with DMD.)
Someone also just sold a massive amount of $80 May puts. They expect good news it would seem.
How did lobbing insults at the female population--by way of insulting a man suggesting that he's "a little girl", as if being a girl is something to be ashamed of--become part of this discussion? Pretty sure the likes of Jenn and Christine sort of kill your notion that females are weak-minded, and I'd wager they've done far more for DMD than you have. An argument is one thing, but the need to insult doesn't make you look all that bright.
I'll get off my soapbox now. I guess I just find it odd that one would take such an approach in their argument when two females are the main reason SRPT even had a shot at AA in the first place.
Yep. That's how I read the press release as well. So just exactly what is SRPT supposed to do with the P3 trial design then?
I can't help but think there was some heavy-handed #$%$ going on here with GSK involved.
And I do wonder if a buyout is now a real possibility.
With the blow dealt from the FDA, in the very least, the company needs to get an agreement with GSK/RNA to get eteplirsen rights there. Given what it sounds like the FDA will be expecting in regards to a Phase 3, we are going to have a nearly impossible time gathering enough exon-51 boys that fall within the required parameters (age, walking distance) for this test from the United States alone. (We don't want to recruit too young like RNA did as that will lead to skewed results.) And we can't recruit boys from Europe because of the RNA patent. Without getting boys from Europe, I think we're looking at 2-3 years for a U.S. only trial.
Like a few others have posted in the past few days, my biggest concern with Z-160 is the dropout rate, particularly with the placebo group in the LSR trial. One poster astutely pointed out that patients enrolling in this trial are likely patients who very specifically don't want to take opiods. Thus, they are more likely to complete the trial. I agree with that, but even so, pain is pain.
There's no doubt that a significant number of the placebo LSR patients would not be able to live with pain for 6 entire weeks. So I've been worried about how the trial will account for this. I went onto the clinicaltrials website to do some digging. I discovered that patients in the trial are allowed to take rescue medications. A rescue medication is a quick-relief or fast-acting medication patients in clinical trials may be given besides the investigational drug or control that can alleviate symptoms due to disease or lack of efficacy of the study treatment. It acts quickly to stop symptoms, but the effects are not long lasting.
On top of that, the use of rescue medications is one of the secondary endpoints. Pretty safe to assume the placebo group will be using these meds heavily and thusly, this should reduce the dropout rate quite a bit. Another pertinent point is that patients cannot test positive for drug use, and alcoholics are excluded. So in other words, the only medication that could be used for pain relief would be the rescue medication.
Feeling a little more confident after learning about all of this.
The reverse split and the results are in absolutely no way related. The negative effects of a reverse split would be strictly psychological; it wouldn't be an indicator of P2 success or failure.
Secondly, your scenario regarding a leak and what it means if one does or does not occur is also bunk.
I swear, some of you completely lack logic. Remove emotion from the equation. This is a huge gamble here, but don't try and connect dots that don't exist.